So what: BioMarin said patients with Morquio syndrome -- a rare genetic disorder that causes skeletal dysplasia and abnormal breathing -- saw improved breathing and walking after 36 to 48 weeks on GALNS, triggering plenty of analyst optimism over the drug's approval. With about 3,000 GALNS patients worldwide representing a market opportunity of roughly $500 million to $800 million, the positive results also reinforce BioMarin as a particularly tasty takeover candidate.

Now what: Management now plans to submit marketing applications for GALNS starting in the first quarter of 2013. "The GALNS clinical program is currently the highest development priority at BioMarin, and this positive Phase 3 study serves as a potentially transformative milestone for the company," said CEO Jean-Jacques Bienaime. So while BioMarin remains too speculative for average investors, today's ultra-encouraging data might make the stock an interesting pick for biotech-savvy Fools.